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Article

VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy.

Journal of translational medicine - 8 Jan 2022

Cheng Cheng, Weiss Lan, Leinonen Henri, Shmara Alyaa, Yin Hong Z, Ton Timothy, Do Annie, Lee Jonathan, Ta Lac, Mohanty Eshanee, Vargas Jesse, Weiss John, Palczewski Krzysztof, Kimonis Virginia

Abstract excerpt

BACKGROUND: Pathogenic gain of function variants in Valosin-containing protein (VCP) cause a unique disease characterized by inclusion body myopathy with early-onset Paget disease of bone and frontotemporal dementia (also known as Multisystem proteinopathy (MSP)). Previous studies in drosophila models of VCP disease indicate treatment with VCP inhibitors mitigates disease pathology. Earlier-generation VCP...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 34998409
DOI: 10.1186/s12967-021-03186-6
PMCID: PMC8742393

Genes

VCP

Variants

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Related research

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Research trail→

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VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy.PMID 34998409Read ↗

P97/VCP ATPase inhibitors can rescue p97 mutation-linked motor neuron degenerationSemantic proximity Read ↗

Transgenic mice expressing mutant forms VCP/p97 recapitulate the full spectrum of IBMPFD including degeneration in muscle, brain and boneSemantic proximity Read ↗

A progressive translational mouse model of human valosin‐containing protein disease: The <i>VCP</i><sup>R155H/+</sup> mouseSemantic proximity Read ↗

Transgenic expression of inclusion body myopathy associated mutant p97/VCP causes weakness and ubiquitinated protein inclusions in miceSemantic proximity Read ↗

VCP activator reverses nuclear proteostasis defects and enhances TDP-43 aggregate clearance in multisystem proteinopathy modelsSemantic proximity Read ↗

The Homozygote VCPR155H/R155H Mouse Model Exhibits Accelerated Human VCP-Associated Disease PathologySemantic proximity Read ↗

Novel VCP activator reverses multisystem proteinopathy nuclear proteostasis defects and enhances TDP-43 aggregate clearanceSemantic proximity Read ↗

Valosin-containing protein (VCP) is required for autophagy and is disrupted in VCP disease.Semantic proximity Read ↗

The multiple faces of valosin-containing protein-associated diseases: inclusion body myopathy with Paget's disease of bone, frontotemporal dementia, and amyotrophic lateral sclerosis.Semantic proximity Read ↗

IBMPFD Disease-Causing Mutant VCP/p97 Proteins Are Targets of Autophagic-Lysosomal Degradation.Semantic proximity Read ↗

Skeletal muscle cell protein dysregulation highlights the pathogenesis mechanism of myopathy-associated p97/VCP R155H mutations.Semantic proximity Read ↗

Pathological consequences of VCP mutations on human striated muscle.Semantic proximity Read ↗

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